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Jane E. Henney, Ph.D - Commonwealth Club of California

"This text contains Dr. Henney's prepared remarks. It should be used with the understanding that some material may have been added or deleted during actual delivery."

Remarks by:
Jane E. Henney, M.D.

Commissioner of Food and Drugs
U.S. Food and Drug Administration

"FDA: Leading the World in Public Health"
Commonwealth Club of California San Jose
August 3, 2000

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A few weeks ago I was privileged to be among the audience in the East Room of the White House when President Clinton and British Prime Minister Tony Blair announced the successful mapping of the human genome. The President declared this achievement the "first draft of the book of life," and compared it to new frontiers discovered by Lewis and Clark.

Francis Collins, director of the National Human Genome Research Institute at NIH, and head of the publicly funded international effort known as the Human Genome Project; and Craig Venter, CEO of the biotechnology company, Celera Genomics, in Rockville, Maryland, represented the public and private sectors involved in this years long, sometimes rough and tumble, effort to map the human genome. Each presented their views of the meaning of the achievement and provided me with much on which to reflect.

Completion of the "rough draft" has been compared to Gregor Mendel's establishing the field of genetics, and Watson and Crick's discovery of the double helix structure of DNA. This event has brought us, without a doubt, to the threshold of a frontier that promises much for us and future generations.

However, even while acknowledging this major achievement, we need to remind ourselves that this is a map, not an operating manual. It identifies all the parts but we don't yet know what the parts do, or how to assemble them, or how precisely to turn the on and off switches. Maps are useful, for they orient us...and give us a sense of measurement. Yet, as anyone who's spent time pouring over a topographical map can attest, sometimes it's useful to look up and read the landscape as well. Maps don't interpret or imagine...and we know that this map is still a rough draft, with much to be understood before a logical itinerary can be created for a productive journey.

So while some of these gaps are being filled in, we still have time for a collective pause for reflection and dialogue on the impact of the human genome-and how this knowledge will be used.

For there is little question that the mapping of the human genome will have a significant impact on all of our lives. In time, this achievement could transform the diagnosis, treatment, and even prevention of diseases that today are still crippling many in our society. It's conceivable that diseases like Alzheimer's, cancer, and diabetes will be treatable if not curable sooner than may well have been imagined only a few years ago.

As we consider how to use this new knowledge, we must balance the medical benefits of this discovery with our basic beliefs and values. As Albert Einstein once said, " A little knowledge is a dangerous thing...so is a lot."

However, during the limited time I have to spend with you I will focus on my views on how this regulatory agency will carry out its mission of protecting and promoting the public health in reviewing the many products that will no doubt be an outgrowth of the human genome project.

I'm sure you're familiar with the FDA. Many of you here today make it your business to know what FDA does and how we do our job. You may be surprised to know that a recent survey shows that seventy percent of Americans can name, unprompted, FDA as the federal agency responsible for reviewing new medical products for the marketplace. Most people can remember reading a story in the newspaper or watching a spot on the nightly news about FDA issues-a new drug available on the market...a tampering...or a food that's been recalled.

As the nation's largest consumer protection and public health agency--with a history dating back to 1906--the FDA oversees an immense range of products, including food, cosmetics, veterinary drugs and feeds, vaccines, the nation's blood supply, and the most complex drugs and medical devices. Twenty-five percent of the U.S. Gross Domestic Product is under the purview of the FDA.

The agency conducts its business on behalf of the public with very limited resources, a little over a penny a day per person. It does this by first attending to those issues that pose the greatest risk, while also maintaining the agility to meet the unexpected challenges that crop up.

FDA has earned the trust of those it serves. An April survey of federal regulatory agencies, conducted by the Pew Charitable Trust, asked the medical profession, patient advocates, the chronically ill, and those business regulatory officers whom we interact with, their opinions about the FDA and its decision-making.

First and foremost, these highly varied groups have confidence in the agency. Seventy-two to eighty-five percent of these groups trust FDA to make the right decisions, and seventy-four to eighty-seven believe that FDA uses good science in its decision-making. The message is simple and it came across loud and clear. Trust and confidence are earned by relying on science, not whim.

When the FDA decides whether to permit a new drug or vaccine to be marketed, whether to recall a food product, or whether to issue a warning about how a medical device can fail, the agency's judgment has to be firmly grounded in the best science available. Most Americans aren't scientists, but they know intuitively how important science is in making regulatory decisions. They trust FDA to employ scientific principles, methods and data to drive its policy-making as well as the tough decisions on the relative risk and benefits of products under its jurisdiction.

Maintaining the public trust and confidence will be essential as we wrestle with the kinds of issues that arise from mapping the human genome. But the real challenge is that we'll need to do it in an environment that's been forever changed by the human genome project. For like never before in our history, the wheels of discovery will likely turn faster than our society's ability to incorporate the impact of the vast array of products and span of possibilities that knowledge from the human genome project will produce.

There are already questions of ethics and values--not just science--such as the issue of privacy, that have been posed. A recent Time/CNN poll showed that forty-seven percent of respondents said they think it's morally wrong to develop technology to map the genome. Seventy-five percent said they would not want their health insurers to know what diseases they were predisposed to get, and eighty-four percent said they would not want the government to have this information. Interestingly, thirty-five percent said they would not even want to know themselves what genetic diseases they might be predisposed to.

Public policy is already being shaped by these concerns. President Clinton has signed an executive order prohibiting every federal department and agency from using genetic information in any hiring or promotion activity. He's also endorsed legislation that was introduced to extend the same protections to the private sector, and to individuals buying health insurance.

Other important questions beyond privacy have already begun to confront clinicians and potential patients as well.

Let me give you some examples.

Two breast cancer susceptibility genes, BRCA1 and BRCA2, have been identified. Mutations in these genes are thought to account for between five and ten percent of all breast cancer cases. It's now possible to detect mutations in these two genes, and this scientific advance presents a dilemma.

On the one hand, it may be important to some women to know they have this increased risk for developing breast cancer. On the other hand, what are women to do with this knowledge, given that it only accounts for five to ten percent of the total susceptibility factors? Should drugs that have their own inherent risks be recommended to prevent cancer? Should a woman have preventive surgery?

Clearly these are not factors that can viewed in isolation but do contribute to an increasingly complex clinical picture.

Genetic tests are already under development to predict a person's susceptibility to diseases for which there are no definitive treatments. Alzheimer's Disease and Huntington's Chorea are two examples where the disease remains essentially untreatable at this point, raising serious questions about the benefit of conducting such tests.

There is another area of promise that will likely yield results long before we see widespread use of genetics-based medical therapies. The knowledge from the human genome project and other new technology will likely be used to determine how to treat a particular patient. What drug to use, if any, and how much?

A very promising diagnostic tool in this regard that could help physicians select proper drugs for individual patients, is DNA chip technology. This technology rapidly determines how a patient will metabolize and eliminate a drug, making it possible for physicians to know whether a certain drug will work in a particular patient at the standard dose, or whether the dose needs to be adjusted up or down.

It's entirely conceivable that one day a physician will be able to choose a treatment and tailor the dose to fit the metabolic profile of an individual patient, much more effectively than this can be done today.

As we gain more information about the human genome, the interest in--and the resources devoted to--gene-based therapeutics increases as well. Gene therapy studies are examining a broad range of potential treatments, including stimulating the body's immune reaction to tumors, and stopping the replication of HIV in AIDS patients. There's currently renewed emphasis on gene therapies for genetic diseases such as hemophilia A and B, and cystic fibrosis.

These are but some of the coming attractions resulting from the very beginning of knowledge coming from the human genome project.

What are the challenges for FDA in this rapidly evolving picture? Our challenges are not new, but the learning curve gets a bit steeper with each new discovery. The amount of information being generated is staggering and the science is becoming increasingly complex, which results in our first challenge.

We simply must maintain the strongest possible science base. As I mentioned earlier, public trust and confidence in our agency have been very tightly linked to our ability to make decisions grounded in science. FDA must have the scientific expertise to adequately assess the safety and effectiveness of these novel products and technologies. The agency must be equipped to make the judgements about which new products should enter the marketplace. If we're not prepared, our decision-making will become increasingly risk averse--which could slow the process of getting needed products to market--or our decisions could be wrong. Neither of these outcomes is acceptable.

To make these critical decisions, our scientists must be current in their fields, quick learners in new areas, and have the ability and creativity to collaborate with other scientists, both within FDA and outside of FDA. Top-notch scientists today are looking for career opportunities that will give them the prestige they deserve, and time to think and learn, and interact with their colleagues. We in government must be able to compete for their services with institutions in the public and private sector.

It should go without saying that substantial resources are needed to do this. Our capacity to stay abreast of new technology in the future depends on our having adequate funding for both salary and support to keep our scientists at the top of their game. If this is not done, our decisions-and the nation's trust in the FDA--and consumer confidence in the products developed by the industries we regulate, are at risk.

I also feel compelled to note...as a former drug monitor, clinical investigator, and academic leader, that while it's imperative that FDA earns and keeps the trust of the public, so too is it vital that the scientific and clinical research communities have the same vote of confidence. Discovery cannot result in products if no patients are willing to participate in research studies.

Trust is a fragile thing. As philosopher Joseph Hall said, "A reputation once broken may possibly be repaired, but the world will always keep their eyes on the spot where the crack was."

The public's trust in new technology-and in those that develop it-has recently been put at great risk when research institutions failed to adhere to the standards of good clinical practice, and didn't adequately inform human volunteers about the risks of participating in clinical trials.

Government and academic institutions are responding to this crisis of confidence, but assuring that biomedical research-from the bench to the bedside--is conducted ethically and responsibly will need continued vigilance.

What does all this mean in terms of our approach to regulating products that may result from the growing body of genetic information? Simply this-we intend to use the same principles we use in all of our regulatory decisions.

Science and risk-based analyses to evaluate the safety and effectiveness of products, with open and transparent processes used to provide us with input from the public.

There's nothing new or glamorous to that approach, but it will assure an orderly procession of safe and effective products to the marketplace.

So no matter how scientifically sophisticated or novel these new products may be, we'll continue to be vigilant at every stage of the process of bringing them to the marketplace-just as we've always been.

Before I close, I'd like to add another caveat or placeholder for your consideration. And that is that while products developed from the knowledge embedded in the human genome hold great promise for many diseases, we must not lose sight of the public health problems confronting the nation right now. Some of these diseases and conditions may eventually be addressed through genetic research and development, but many are problems demanding our immediate attention.

Conditions like childhood asthma and obesity, that are becoming more prevalent and are a real threat to our nation's health and well-being. Diseases like tuberculosis that are increasing, with causative organisms that are becoming resistant to antibiotics.

Our nation's research and subsequent product portfolio should be sufficiently diversified to assure that we have emphasis not only on molecular approaches to understanding disease and treatment but also on more traditional approaches to diseases where there is insufficient knowledge to design an approach at the molecular level.

These are astonishing times in science and medicine. In times past, scientific discovery proceeded at a pace that gave society time to digest new information, look at it from all sides, and gradually work it into the way people viewed the world. Today, science and the technologies that result from it are changing at such a fast pace that it's difficult for the human mind to grasp their potential promise and pitfalls before they are upon us.

A magazine writer expressed it this way: he wrote, "Science empties its discoveries on you so fast that you stagger beneath them in hopeless bewilderment...Human nature can't endure much more." The magazine was The Atlantic Journal, and believe it or not, the year was 1833. Those words are still relevant today.

With the astonishment at advancing knowledge comes excitement, and this is an exciting time to be at the FDA. The challenges are immense, but the potential rewards of service, and being part of improving the health and well-being of our citizens in terms of protecting and promoting health, are equally immense.

As new knowledge about the genetic basis of human disease is coming to light, the American people are trusting the FDA to make sure that the products of this research are safe and effective, just as it's always done.

A useful metaphor might be the "Regulator" clock--that classic American timepiece that hung on the walls of schoolhouses, depots and other public places in the 19th century and beyond, separating hour from hour, creating order, and establishing a rhythm. In this sense, FDA provides a timely and predictable process for bringing products to market with the assurance that approval decisions have been grounded in the very best science.

To end with the words of one our greatest innovators, "Creating a new theory is not like destroying an old barn and erecting a skyscraper in its place. It is rather like climbing a mountain, gaining new and wider views, and discovering unexpected connections between our starting point and its rich environment."

As Albert Einstein imagined, science will continue to widen our possibilities. As a science-based regulatory agency, the FDA must remain committed to the fundamental principles on which sound regulation is always based - fairness, predictability, integrity, and strong science.